Experimental painkiller blocks GPCR signaling inside the cell
Researchers have developed a new highly effective therapeutic for pain relief by altering the chemical properties of an antinausea drug, netupitant. The modified drug is able to enter the intracellular membrane-bound endosome and target the GPCRs therein, rather than at the cell surface, that leads to optimal pain relief. The study, published in the Proceedings of the National Academy of Sciences (PNAS) on May 22, 2023, was led by Nigel Bunnett, Professor and Chair of Molecular Pathobiology at NYU College of Dentistry, and illustrates how GPCR-mediated pain signaling occurs inside the endosomes rather than at the surface, highlighting the need for drugs that can reach receptors within the cells itself.

Inhibition of YTHDF2 results in a more effective and safe radiotherapy
In a study published in Cancer Cell on May 25, 2023, researchers from the University of Chicago and colleagues reported that the inhibition of YTHDF2, an immune suppressor protein, can be a valuable strategy to improve radiotherapy outcomes by overcoming resistance while enabling extra help from the immune system.

Experimental painkiller blocks GPCR signaling inside the cell
Researchers have developed a new highly effective therapeutic for pain relief by altering the chemical properties of an antinausea drug, netupitant. The modified drug is able to enter the intracellular membrane-bound endosome and target the GPCRs therein, rather than at the cell surface, that leads to optimal pain relief. The study, published in the Proceedings of the National Academy of Sciences (PNAS) on May 22, 2023, was led by Nigel Bunnett, Professor and Chair of Molecular Pathobiology at NYU College of Dentistry, and illustrates how GPCR-mediated pain signaling occurs inside the endosomes rather than at the surface, highlighting the need for drugs that can reach receptors within the cells itself.
Inhibition of YTHDF2 results in a more effective and safe radiotherapy
In a study published in Cancer Cell on May 25, 2023, researchers from the University of Chicago and colleagues reported that the inhibition of YTHDF2, an immune suppressor protein, can be a valuable strategy to improve radiotherapy outcomes by overcoming resistance while enabling extra help from the immune system.
Other news to note for May 26, 2023
Additional early-stage research and drug discovery news in brief.
PAK4 inhibitor from Hyperway Pharmaceutical has improved safety and PK profile
Work at Hyperway Pharmaceutical Co. Ltd. has led to the discovery of a novel PAK4 inhibitor...
Hanall and Daewoong partner with Nurron to advance compounds for neurodegenerative disorders
Hanall Biopharma Co. Ltd. and Daewoong Pharmaceutical Co. Ltd. have entered into...
Bright Angel Therapeutics patent describes new Hsp90 inhibitors
Bright Angel Therapeutics Inc. has patented heat shock protein 90 inhibitors reported to be useful...
Psilera nominates non-hallucinogenic psilocybin derivative as lead clinical candidate
Psilera Inc. has selected a novel, non-hallucinogenic psilocybin derivative, PSIL-006...
MyoDys45-55 restores dystrophin and improves functional measures in murine model of DMD
Myogene Bio LLC has developed an approach through CRISPR7Cas9 technology...
Teon Therapeutics identifies new cannabinoid CB2 receptor modulators for cancer
Teon Therapeutics Inc. has reported 4-hydroxy-2-oxo-1,2-dihydro-1,8-naphthyridine-3-carboxamide...

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Conferences

Next-generation gene therapy shows superior efficacy for spinal muscular atrophy
Neurology/Psychiatric
Spinal muscular atrophy (SMA) is caused by mutations in the SMN1 gene, which encodes survival motor neuron 1, leading to reduced protein expression levels and degeneration of motor neurons in the spinal cord, with the consequent muscle atrophy. There is thus a need for new AAV gene therapies for...
SKG-0106 shows durable efficacy in preclinical models of neovascular AMD
Ocular
Researchers from Skyline Therapeutics (Shanghai) Co. Ltd. presented preclinical data for the new recombinant AAV vector therapeutic SKG-0106, being developed for the treatment of neovascular (wet) age-related macular degeneration (AMD).
AAV9-GLA restores α-galactosidase levels in murine model of Fabry disease
Endocrine/Metabolic
Fabry disease is a metabolic disease characterized by a deficiency in the lysosomal α-galactosidase enzyme caused by mutations in the GLA gene. This leads to substrate accumulation in the lysosomes, cellular dysfunction and organ damage.
NTX-1175 channel blocker is effective in visceral pain models
Gastrointestinal
Patients with irritable bowel syndrome (IBS) have chronic abdominal pain as a result of visceral hypersensitivity. Voltage-gated sodium channels control cellular excitability and are involved in the pathophysiology of several functional gastrointestinal disorders. Researchers from Nocion...

Cancer

Discovery of new IDO-1 inhibitors with potent anticancer activity
Researchers from Guilin Medical University (GLMU) reported the discovery and preclinical evaluation of novel indoleamine 2,3-dioxygenase 1 (IDO-1) inhibitors as potential anticancer agents. Synthesis and optimization of a series of chromone-oxime derivatives containing piperazine sulfonamide...

Infection

AAV9-based therapy neutralizes rabies viral infection in neurons
Conferences
Rabies virus is among the most neurotrophic known virus and it has a fatality rate of almost 100%. There is a need for antibody-induced protection in the central nervous system, since rabies virus can cross the blood-brain barrier but antibodies cannot. Investigators from Auburn University and the...

Westvac announces NMPA clearance for clinical trials of bivalent and trivalent COVID-19 protein vaccines
Immune
China's National Medical Products Administration (NMPA) has given drug clinical trial approval for two new COVID-19 vaccines against the current XBB variants developed by Westvac Biopharma Co. Ltd. and West China Medical Center at Sichuan University.
Assembly Biosciences patents new compounds for hepatitis B
Patents
Assembly Biosciences Inc. has disclosed heteroaryl carboxamide derivatives reported to be useful...

Neurology/Psychiatric

Let there be walk: Onward Medical sees first use of movement-restoring lead
Artificial intelligence
The researchers who enabled patients with spinal cord injuries to walk independently after implanting programmable electrodes below their lesions have now taken things one step further, restoring direct communication from the brain to the spinal cord, enabling the brain rather than an external...
Gene expression study gives clues to pain insensitivity syndrome
Researchers have used cell culture experiments to understand how gene expression was affected in a patient with a rare pain insensitivity syndrome, and have identified a network of hundreds of genes whose expression was changed compared to sex-matched controls. Published online in the journal Brain...
AEG-1 regulates granule cell dispersion, seizure development in model of temporal lobe epilepsy
Conferences
Researchers from Kyungpook National University presented data from a study that aimed to investigate the endogenous mechanisms involved in granule cell dispersion (GCD) and its role in epileptic seizures in temporal lobe epilepsy (TLE).
Where does it hurt? For chronic pain, in the orbitofrontal cortex
Long-term brain recordings from four patients with chronic pain have led investigators at the University of California at San Francisco to identify brain signals that could serve as biomarkers for each individual patients’ pain. The study, which was published online in Nature Neuroscience on May...
Merck Sharp & Dohme reports RIPK1 inhibitors for ALS and inflammatory disorders
Patents
Research at Merck Sharp & Dohme Corp. has led to the development of receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis (ALS) and inflammatory disorders.

Endocrine/Metabolic

Bloomsbury Genetic’s liver-targeted AAV-LK03 gene therapy BGT-OTCD cleared to enter clinic
Gene therapy
The U.K. Medicines and Healthcare Products Regulatory Agency (MHRA) and the Gene Therapy Advisory Committee (GTAC) have approved a clinical trial application (CTA) submitted by University College London (UCL) to initiate a phase I/II trial of BGT-OTCD, Bloomsbury Genetic Therapies Ltd.’s...

Biomarkers

Low miR-143 expression and high MSI2 expression are associated with poor prognosis in AML
Cancer
Researchers from Wenzhou Medical University and affiliated organizations have published data from a study that aimed to investigate the relationship between the tumor suppressor microRNA-143 (miR-143) and RNA-binding protein Musashi homolog 2 (MSI2), the abnormal expression of which has been...

SDC4 confers poor survival in gastric cancer
Cancer
Gastric cancer persists as the fourth leading cause of cancer-related deaths. Syndecans (SDCs) are a family of four transmembrane heparan sulfate proteoglycans involved in cell proliferation, migration and adhesion, among others, and syndecan-4 (SDC4) expression has been shown to be up-regulated...
SNP in miR-146a is a prognostic marker in acute coronary syndrome
Cardiovascular
Despite advances in diagnosis and treatment, cardiovascular disease remains the leading cause of death worldwide. Single-nucleotide polymorphisms (SNPs) in microRNAs are known to play important roles in acute coronary syndrome (ACS). MicroRNA 146a (miR-146a) is significantly upregulated in human...

Gastrointestinal

Small-molecule AXIN stabilizer from University of Maryland inhibits liver fibrosis
Conferences
Researchers from the University of Maryland presented preclinical data for YA-6060, a novel small molecule showing dual activities of Wnt inhibition and AMPK activation via the mechanism of AXIN stabilization. Since both Wnt/β-catenin and AMPK...
Insilico Medicine identifies new PHD2 inhibitors
Patents
An Insilico Medicine IP Ltd. patent describes Egl nine homolog 1 (EGLN1; HPH-2; PHD2) inhibitors reported to be useful for the treatment of ulcerative colitis and Crohn’s disease.
LPA1 receptor agonist improves NASH-related outcomes in preclinical models
Conferences
Currently, there is no FDA-approved drug for nonalcoholic steatohepatitis (NASH), which has evolved into the second leading cause of liver transplantation in the U.S. Researchers from Children’s Hospital Los Angeles and Epigen Biosciences Inc....
Researchers present data from murine studies of PAM-369, a muscarinic M3 receptor PAM for enteropathy
Conferences
Researchers from Kyushu University and Mochida Pharmaceuticals Co. Ltd. disclosed recent data along with the chemical structure of PAM-369, a novel muscarinic acetylcholine M3 receptor positive allosteric modulator (PAM), being developed for the...
Researchers report discovery of ROR-107, a GPBAR1 agonist and RORγt inverse agonist for IBD
Conferences
Inflammatory bowel disease (IBD) is a chronic, immunologically mediated disorder of the gastrointestinal tract in which tissue damage and sustained inflammation lead to long-term dysfunction of the gastrointestinal tract.
Tackling spores is new strategy for recurrent Clostridioides difficile
Infection
An experimental antibiotic was able to prevent recurrent Clostridioides difficile infections (rCDI) by killing bacterial spores as well as growing spores. Researchers from the University of Notre Dame reported their findings in the Proceedings of...

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